Calliditas Therapeutics AB announced that the US Food and Drug Administration (FDA) has granted full approval for Tarpeyo (budesonide) delayed-release capsules, aimed at diminishing kidney function loss in adults diagnosed with primary immunoglobulin A nephropathy (IgAN) who are susceptible to disease advancement.
Initially receiving accelerated approval in December 2021 based on proteinuria as a surrogate marker, Tarpeyo has now achieved the status of being the first treatment for IgAN to receive full FDA approval predicated on evaluations of kidney function.
“We are thrilled that adult IgAN patients at risk for progression in the US can now have access to this pioneering treatment option that could help preserve their kidney function and, hence, impact the progression of their disease,” said Renee Aguiar-Lucander, CEO of Calliditas, in the company’s press release.
The FDA’s endorsement stems from results obtained from the company’s Phase III NefIgArd clinical trial. This randomized, double-blind, multicenter study evaluated the effectiveness and safety of administering 16 mg of Tarpeyo once daily compared to a placebo, alongside optimized renin-angiotensin system inhibitor (RASi) therapy, in adult patients diagnosed with primary IgAN.
In this study, Tarpeyo demonstrated a confirmed and statistically significant advantage over placebo (p<0.0001) in the estimated glomerular filtration rate (eGFR) throughout a two-year period. This study included nine months of either Tarpeyo with optimized RASi or a placebo and optimized RASi, followed by 15 months without the study drug.
After two years, the Tarpeyo group showed a 6.11 mL/min/1.73 m² reduction in eGFR, whereas the placebo group exhibited a 12.0 mL/min/1.73 m² decrease (p<0.0001). This indicates that Tarpeyo-treated patients experienced 50 percent less kidney function decline than those receiving the placebo over this period.
In this study, the most frequently observed adverse reactions (occurring in ≥5 percent of participants) included peripheral edema, hypertension, muscle spasms, acne, headaches, upper respiratory tract infections, facial edema, weight gain, dyspepsia, dermatitis, arthralgia and an increase in white blood cell count.
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IgAN, a rare kidney disorder, is marked by the accumulation of IgA deposits within the kidneys. This accumulation triggers inflammation and damages kidney tissues, leading to the leakage of blood and protein into the urine. Complications from IgAN may include increased blood pressure and the potential onset of chronic kidney disease, sometimes advancing to kidney failure.
A suggested origin of IgAN involves the hyperactivation of B cells in the ileum or small intestine, resulting in the excessive release of IgA into the blood. This can initiate an autoimmune reaction, culminating in the formation of immune complexes that deposit in the kidneys.
Calliditas formulated Tarpeyo as an immunosuppressant aimed at gut B cells. As an oral formulation of budesonide, Tarpeyo falls under the corticosteroids category, known for their anti-inflammatory properties.
Engineered for delayed release, Tarpeyo is designed to maintain its structure until it arrives at the ileum of the digestive tract. There, it specifically targets the mucosal B cells in the ileum to inhibit antibody formation.
Related: Filspari (Sparsentan) Rare Kidney Disease Drug Gets FDA Approval
Availability and Price of Tarpeyo
Tarpeyo is available solely through Biologics by McKesson, Calliditas’ specialty pharmacy. To begin treatment, prescribers must fill out the Tarpeyo Touchpoints Enrollment Form, which acts as the official prescription. This form connects patients with the full array of benefits provided by Tarpeyo Touchpoints, including financial aid programs designed to reduce or completely cover out-of-pocket costs, access to Calliditas’ team of care navigators, pharmacists and nurse educators, and the advantage of convenient at-home, next-day delivery.
Tarpeyo oral delayed-release capsule 4 mg costs around $15,934 for a supply of 120 capsules for cash-paying customers.
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